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The importance of rare disease research, as told by our employees.

According to the National Institutes of Health (NIH), a rare disease is a condition that affects fewer than 200,000 people in the US. However, the rare disease community makes up a far greater number. In fact, the NIH claims that, with more than 7,000 known rare diseases, about 25–30 million individuals across the country live with a rare disease.

Despite a collectively large patient population, individuals living with conditions that fall under this category haven’t always been advocated for. Rare diseases had previously been called “orphan diseases” due to a lack of interest in adopting new treatments. It wasn’t until the Orphan Drug Act of 1983 that financial incentives were distributed to encourage companies to develop new drugs for rare diseases.

Since then, the clinical trial industry has made incredible strides in research, and we at Praxis are proud to continue to work on trials that impact individuals living with rare diseases. As we dedicate our time and efforts toward groundbreaking therapies and treatments, we wanted to share thoughts from our employees, in their own words, as to why awareness around these conditions is so important. Read on to hear what they had to say.

“A rare disease can mean many things. It may be that there isn’t a treatment or cure available on the market yet, which make clinical trials all the more important. It can also mean that doctors and scientists simply need more data and research results to learn and understand the condition – how it affects the body, how it’s contracted, what treatments could impact it, what its long-term effects are, etc. Through research and clinical trials, we’re able to learn more about rare diseases and how modern medicine can help.”
Sara Root, Account Director

“Supporting clinical trials for rare diseases has helped open my eyes to how many ‘rare’ diseases exist and the lack of available treatment options. Rare disease awareness sheds light on this important topic, helping bring forth options for possible relief.”
Maida Tutundzic, Project Manager

“One reason I think rare disease awareness is so important is that it helps provide access to better treatment. When working on a rare disease study for Praxis, we often turn to social listening to learn more about what patients may be feeling. Reading these insights, I’ve been shocked to learn how many rare disease patients have gone to a doctor or an emergency room where the provider is unfamiliar with their condition and, in turn, the patient cannot receive treatment.”
Rosalind Cardone, Senior Copywriter

“Rare disease awareness can help connect patients and their families with others who are experiencing a similar journey. Only someone facing the same situation can truly understand what it feels like.”
Kim Taddeo, Outreach and Events Planner

“Both rare disease patients and their caregivers are hungry for information once they receive a diagnosis.  Praxis understands that awareness around rare diseases not only helps patients and caregivers find information about a condition, but also helps them find potential clinical trials that might be a fit, as well as patient communities or support networks that provide a shared understanding of the condition.”
Nina Taylor, Marketing Strategist

If you’re interested in learning what we do at Praxis, or if you’d like to understand more about our work with rare disease clinical research, feel free to reach out.